Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.
Gene Therapy Corrects Factor VIII Levels in Hemophilia A
Posted on: 7 August 2017, source: Medscape
Patients with hemophilia A who received a gene for coagulation factor VIII (FVIII) packaged in a viral vector had sustained levels of FVIII during 1 year of observation, eliminating spontaneous bleeds[2]. Most of them did not need FVIII infusions even in the case of major trauma or surgery. In a late-breaking abstract session here at the International Society on Thrombosis and Hemostasis 2017 Congress, Dr John Pasi (Barts and the London School of Medicine and Dentistry, Queen Mary University of London, UK) said six out of the seven patients in one dose cohort have achieved normal factor VIII levels.
Patients with hemophilia A who received a gene for coagulation factor VIII (FVIII) packaged in a viral vector had sustained levels of FVIII during 1 year of observation, eliminating spontaneous bleeds[2]. Most of them did not need FVIII infusions even in the case of major trauma or surgery. In a late-breaking abstract session here at the International Society on Thrombosis and Hemostasis 2017 Congress, Dr John Pasi (Barts and the London School of Medicine and Dentistry, Queen Mary University of London, UK) said six out of the seven patients in one dose cohort have achieved normal factor VIII levels.
First gene therapy — 'a true living drug' — on the cusp of FDA approval
Posted on: 14 July 2017, source: Chicago Tribune
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients' revved-up immune cells to fight the disease, concluding that the therapy's benefits for desperately ill children far outweigh its potentially dangerous side effects. The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a new path in the immunotherapy frontier.
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients' revved-up immune cells to fight the disease, concluding that the therapy's benefits for desperately ill children far outweigh its potentially dangerous side effects. The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a new path in the immunotherapy frontier.
South Korea approves first gene therapy drug
Posted on: 12 July 2017, source: Yonhap News
South Korea's health authorities said Wednesday that they have approved the use of the country's first gene therapy drug to help patients with degenerative joint disease. The drug, Invossa, manufactured by local drug firm Kolon Life Science Inc. has obtained the green light from the Ministry of Food and Drug Safety for sales in the domestic market. Invossa is the world's first cell-medicated gene therapy for osteoarthritis, a disorder involving movable joints. The company said the drug can be injected into the joint cavity of a patient. The drug has proven to reduce osteoarthritis pain and improve cartilage through clinical trials, said Kolon Life Science, expecting to help an estimated five million patients in the country.
South Korea's health authorities said Wednesday that they have approved the use of the country's first gene therapy drug to help patients with degenerative joint disease. The drug, Invossa, manufactured by local drug firm Kolon Life Science Inc. has obtained the green light from the Ministry of Food and Drug Safety for sales in the domestic market. Invossa is the world's first cell-medicated gene therapy for osteoarthritis, a disorder involving movable joints. The company said the drug can be injected into the joint cavity of a patient. The drug has proven to reduce osteoarthritis pain and improve cartilage through clinical trials, said Kolon Life Science, expecting to help an estimated five million patients in the country.
Gene Therapy & Mesothelioma
Posted on: 18 June 2017, source: mesothelioma.com
Technological innovations are changing the world we live in every day, including the way society views diseases that have been around for hundreds of years. Cancer continues to be a leading cause of death around the world, but day by day we, as a society, are getting closer to finding a cure. In 2016, former President Barack Obama asked former Vice President Joe Biden to head the Cancer Moonshot Initiative (CMI), whose overarching goal would be to find a cure for cancer by the year 2020. In the time that’s passed, Biden has formed a committee of governmental organizations and a Blue Ribbon Panel that provided recommendations of how to achieve the lofty goals, and set to bringing this moonshot into reality.
Technological innovations are changing the world we live in every day, including the way society views diseases that have been around for hundreds of years. Cancer continues to be a leading cause of death around the world, but day by day we, as a society, are getting closer to finding a cure. In 2016, former President Barack Obama asked former Vice President Joe Biden to head the Cancer Moonshot Initiative (CMI), whose overarching goal would be to find a cure for cancer by the year 2020. In the time that’s passed, Biden has formed a committee of governmental organizations and a Blue Ribbon Panel that provided recommendations of how to achieve the lofty goals, and set to bringing this moonshot into reality.
Altered virus may expand patient recruitment in human gene therapy trials
Posted on: 14 June 2017, source: Science Daily
For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of North Carolina researchers have found a solution that may allow it to evade the body's normal immune response.
For many patients, participating in gene therapy clinical trials isn't an option because their immune system recognizes and fights the helpful virus used for treatment. Now, University of Florida Health and University of North Carolina researchers have found a solution that may allow it to evade the body's normal immune response.
Allergy Breakthrough with Gene Therapy
Posted on: 8 June 2017, source: World Health
It might soon be possible for a single treatment to provide life-long protection against harsh allergies including asthma. An immunology research team at The University of Queensland led by Professor Ray Steptoe has figured out how to disable the immune response that triggers allergic reactions. The research team operates out of the university's Diamantina Institute. Professor Steptoe's lab is situated at the Translational Research Institute. The research was funded by the National Health and Medical Research Council and the Asthma Foundation. The research team's findings are published in JCI Insight.
It might soon be possible for a single treatment to provide life-long protection against harsh allergies including asthma. An immunology research team at The University of Queensland led by Professor Ray Steptoe has figured out how to disable the immune response that triggers allergic reactions. The research team operates out of the university's Diamantina Institute. Professor Steptoe's lab is situated at the Translational Research Institute. The research was funded by the National Health and Medical Research Council and the Asthma Foundation. The research team's findings are published in JCI Insight.
European Child Receives Strimvelis Gene-Therapy Treatment
Posted on: 3 May 2017, source: Rare Disease Report
Before March, only 1 patient had ever been treated via commercial gene therapy. GlaxoSmithKline (GSK) doubled that total with their announcement this week. In 2015, a European patient with familial chylomicromenia received Glybera to restore their LPL enzyme levels. On Tuesday, GSK announced that it treated its first patient in March, almost a full calendar year after their drug, Strimvelis, was approved for sale. Strimvelis is assumed to provide an outright cure for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), a rare disorder caused by the absence of the essential protein adenosine deaminase (ADA). ADA is required to produce lymphocytes. It leaves babies without a fully-functioning immune system and extremely vulnerable to infections.
Before March, only 1 patient had ever been treated via commercial gene therapy. GlaxoSmithKline (GSK) doubled that total with their announcement this week. In 2015, a European patient with familial chylomicromenia received Glybera to restore their LPL enzyme levels. On Tuesday, GSK announced that it treated its first patient in March, almost a full calendar year after their drug, Strimvelis, was approved for sale. Strimvelis is assumed to provide an outright cure for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), a rare disorder caused by the absence of the essential protein adenosine deaminase (ADA). ADA is required to produce lymphocytes. It leaves babies without a fully-functioning immune system and extremely vulnerable to infections.
Gene Therapy News
- Spark's Gene Therapy SPK-8011 to Treat Hemophilia A and B Advance in Clinical Trials - Hemophilia News Today
Mon, 21 Aug 2017 12:12 GMT - Cancer Gene Therapy Market Opportunities, In-Depth Analysis, Challenges and Applications Forecast to 2022 - DailyNewsKs
Mon, 21 Aug 2017 09:22 GMT - YOUR HEALTH Gene therapy for better eyesight - WQAD.com
Fri, 18 Aug 2017 23:28 GMT - Gene Therapy Can Restore Rare Immunodeficiency - The Independent
Fri, 18 Aug 2017 18:19 GMT - Cellectis Starts BPDCN Phase I Trial with Gene-Edited CAR-T Therapy - Genetic Engineering & Biotechnology News
Fri, 18 Aug 2017 13:48 GMT
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Gene Therapy Literature
Scroll through the latest articles in gene therapy scientific journals.
Recent early view article in Journal of Gene Medicine:
Recent early view article in Journal of Gene Medicine: